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Welcome to the CREATIC ATMP newsletter | |
| | | | Welcome to the third edition of the CREATIC ATMP newsletter. This newsletter gives you a window to the rapidly evolving regulatory and ethical landscape for therapies utilizing genes-, cells and tissue. You will find a collection of news on regulatory developments and discussions spiced up with perspectives and analysis – all with a focus on social, legal and ethical issues.
The newsletter is published quarterly, with the next issue being scheduled for August. If you're interested in staying updated on the latest social, legal and ethical
news related to ATMPs, please sign up here. | | | | |
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| | | |  | | | | In this edition of the newsletter you can find:
- The latest ATMP news
- Upcoming events
- The Hospital exemption
- Commentary
- Research Papers
- What's new at CREATIC?
- Previous webinars available online
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|  | | | | In this section you can find the latest news within the ATMP area. | | | | |
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| | | |  | Harmonisation health technology assessments, a slow roll in right direction | | | | |
| On 12 January, the Regulation on Health Technology Assessment (HTAR) became fully applicable. The new regulations have marked a significant shift in how health technology assessments are conducted across the EU. While Health Technology Assessment agencies anticipate that joint efforts and harmonisation will enhance evaluations' efficiency, tangible evidence is taking time to materialise.
Furthermore as implementation rolls out in stages national agencies are navigating local challenges. A representative from the National Agency for Medicines and Medical Devices of Romania has highlighted how adapting internal processes to meet new timelines and requirements demands additional resources and expertise. The State Medicines Control Agency of Lithuania shared the concern that the challenges related to joint clinical assessment connect to the shortage of experienced HTA assessors and, hence, a lack of capacity.
You can read more here.
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| | | |  | Warning - Unregulated cell therapies are being sold in Europe | | | | |
| EMA has issued a warning to the public about the dangers of unregulated ATMP's that are being offered illegally to patients in Europe. Unregulated advanced therapy medicinal products pose serious risks to health. According to EMA a number of individuals, companies and clinics have been marketing unregulated ATMPs directly to patients.
Read more here.
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| A new interactive clinical trial map is now accessible from the public website of the Clinical Trials Information System. The map provides patients and healthcare professionals with easy access to comprehensive, real-time information about clinical trial conducted in the EU, increasing access to clinical research in the European Union.This makes it easier to identify clinical trials that may be relevant for patients to participate in.
Read more here.
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| .png) | Four ways modular cleanroom design supports the ATMP revolution | |
| | | | ATMP manufacturing requires stringent cleanroom controls that lives up to GMP standard. Due to the operational challenges and potential manual processing steps in cell and gene therapy production, ATMPs introduce unique cleanroom design requirements. ATMP developers and manufacturers are therefore under increasing pressure to ensure their cleanroom facilities meet these complex needs. In the following article you can read about how the four ways modular cleanroom design supports the ATMP revolution and offers a solution to the with traditional stick-built cleanrooms.
Read more here. | | | | |
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| | | |  | Europe Leads in Cancer-Related Startups, but Faces Scaling Challenges | |
| | | | A new study from EPO on cancer-related technologies, covering the period from 2010 to 2021, show that Europe is losing ground to the U.S. and China, with a decline in patent applications. The study furthermore shows that despite Europe having a higher number of early-stage startups, the U.S. is significantly more successful in scaling them to later growth stages.
Read more here. | | | | |
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| | | | .png) | Why are there only 20 cell and gene therapies in Europe? | |
| | | | In 2020 only 11 cell and gene therapies were available in Europe. Now four years later, there are 20. This seems like a big improvement, but how does it look when we look overseas?
The U.S. market currently counts 43 cell and gene therapies. It seems Europe is lagging behind the U.S, what'sthe reason behind this and how has the situation evolved in the last few years?
Read more here. | | | | |
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| | | | .png) | Comparison of regulatory pathways for the approval of ATMPs in the EU and US | |
| | | | Regulatory agencies in the European Union (EU) and in the US have adapted and launched regulatory pathways to accelerate patient access to innovative therapies, such as advanced therapy medicinal products (ATMPs). But how are the regulatory pathways for the approval of advanced therapies in the European Union and the United States different and similar?
Read more here. | | | | |
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New EMA Guideline relevant for developers and researchers of ATMPS | | | | |
| The European Medicines Agency (EMA) has adopted a new guideline for investigational advanced therapy medicinal products (ATMPs), on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials.
The guideline becomes effective on 1 July 2025 and provides guidance on the structure and data requirements for a clinical trial application for exploratory and confirmatory trials with advanced therapy investigational medicinal products.
This guideline serves as an important guide for developers and researchers to ensure the safety and efficacy of ATMPs during clinical trials.
Read more here.
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Amgros surveillance of alternative agreements | | | | |
| Amgros, the Danish company that secure medicines supplies for Danish public hospitals, has just announced that they are making their data regarding alternative agreements available to the public. According to the data, Amgros has entered alternative agreements with 10 out of a total of 37 requests from suppliers for alternative agreement.
Read more here.
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| | | | | | | Launch of new guidebook for ATMP developers
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| Centre for Future Affordable & Sustainable Therapy Development (FAST) has launched a renewed and expanded guidebook for developing Advanced Therapy Medicinal Products (ATMPs) in the Netherlands. It contains guidance on European and Dutch regulations and provides a roadmap to navigate the complex ATMP development process. Find it here. | | | | | | | |
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|  | | Webinar | 03-06-2025 | |
| | Conference | 26-06-2025 to 27-06-2025 | |
| | Conference | 17-03-2026 to 18-03-2026 | |
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|  | | | | Article 28(2) of the ATMP Regulation (EC) No 1394/2007 introduces the Hospital Exemption. It is a regulated pathway that allows the use of ATMPs without marketing authorisation within the European Union under restricted conditions overseen by national medicine agencies. You can read more about the Hospital Exemption here. The following section delves into the different aspects of the Hospital Exemption. | | | | |
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| | | |  | On November 22, 2024, The CREATIC team held the first CREATIC LabtoP talk about bringing ATMPs from lab to patients. You can now listen to the podcast from the webinar and learn more about bringing ATMPs from lab to patients, with focus on the Hospital Exemption as a pathway.
Whether you're a healthcare professional, researcher, policymaker, or simply interested in cutting-edge medical advancements, this session will provide important perspectives on how hospital exemptions and ATMPs may shape the future of medical innovation and collaboration across sectors.
The conversation took place between Jakob Wested, PhD, associate professor, in Center for Advanced Studies in Bioscience Innovation Law (CeBIL) and PI in the CREATIC project, and Dr. Inge Marie Svane, Professor in Clinical cancer immune therapy and director of the translational research centre, Center for Cancer Immune Therapy (CCIT) at Herlev Hospital in Denmark.
You can listen to the webinar here.
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EU hospital exemption for ATMPs - Patient access and ethical considerations | | | | |
| The Committee on the Ethics of Cell and Gene Therapy
of the International Society for Cell & Gene Therapy reflects on the
ethical issues concerning HE application from the perspective of the patient,
including risk–benefit balance, accessibility and transparency.
Read more here. | | | | |
| | | | | | | Study on Hospital Exemption for ATMPs in Selected EU Countries
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| Different challenges arise with the Hospital exemption. One is how the ATMP regulation leaves the national authorities with some discretion in the decisions regarding the exemption, which has led to the mechanisms being applied quite differently among the Member States. This report explores in depth the implementation of the HE into practice in six different countries (DE, FR, NL, ES and SE, as well as UK).
Read more here. | | | | |
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The Proposed Pharmaceutical legislation and Hospital Exemption | |
| | | | In April 2023, European
commission adopted a proposal for a new Directive and a new Regulation, which
revise and replace the existing general pharmaceutical legislation. In
the proposal the Commission introduced requirements for annual collation,
reporting and reviewing of data by competent national authorities and
publication by the European Medicines Agency (EMA) in a central repository for
the use of the Hospital exemption. The Commission's proposal can be found here.
The European Parliament has on 10 April 2024 adopted its position on the Commission proposal to reform the Pharmaceutical Legislation. The Parliament adopted position can be found here and here. A key takeaway from the
amendments is both the expansion of the Hospital Exemptions scope, but also
additional requirements. The Parliament has introduced the requirement of a
"special need" of an individual patient for a hospital exemption to
satisfy the criteria of 'non-routine basis'. Another requirement is that
applications for a hospital exemption approval will have to provide evidence on
quality, safety and expected efficacy of the ATMPs prepared under the
exemption.
Other than the additional
requirements, the Parliament amendments, also significantly expanded the scope
of the hospital exemption for ATMPs. One example is the inclusion of
"cross-border exchange" of ATMPs in cases of "absence of other
solutions for the individual patient" - something that has not been
possible until now. This amendment contrasts with the original national scope of the exemption.
You can read more about the key takeaways from Parliament's amendments here. | | | | | | | |
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Clearing the way for academically developed ATMP's
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| | | | Decentralised production of academic ATMP's will help addressing high
unmet medical needs in paediatric cancer and other ultra-rare diseases. This
should be on the table in theongoing negotiations on the EU pharmaceutical package.
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| | | | By Jakob Wested,
Associate professor of Pharmaceutical law, Center for advanced studies in
bioscience innovation law (CeBIL) & CREATIC
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| | | | Enabling
decentralized manufacture of ATMP's without a marketing authorization would
significantly improve the access to treatment for the high unmet medical need in
paediatric cancer and other ultra-rare diseases. However, in the
current draft of the proposal
for a new EU pharmaceutical legislation, decentralised manufacturing is only
mentioned as a possibility for ATMPs with a
marketing authorization. This will de facto place academically developed ATMP's
used in clinical trial or administered under the hospital exemption at a
significant disadvantage in an area where commercial development of treatment
is non-existent, insufficient or prohibitively costly.
These days
the EU pharmaceutical package is the topic of meetings and negotiations in
Bruxelles, establishing the regulatory framework for medical development the
next couple of decades. Addressing high unmet medical needs is a priority area
in the proposal, and ATMP's are one of the most promising areas of medical technology to address these needs. However, the current regulatory
framework is designed to accommodate large scale, centralized manufacture, and
not the decentralized and highly personalized production of ATMPs. The EU
pharmaceutical package therefore introduce decentralized manufacturing to
address these challenges specific to ATMP's. But - in the current wording -
only for products with a marketing authorization.
Treatments
for ultra-rare diseases are often without commercial interest even with the
extra market exclusivity available to treatments developed for rare diseases.
Commercial actors have withdrawn existing treatments due to commercial reasons.
Academically developed treatments provide a feasible and necessary complement
to commercially developed ATMPs, in particular in the domain of ultra-rare
diseases. The revision of the EU pharmaceutical legislation provides an
important possibility to introduce features such as decentralized manufacture
that supports this important and life-saving mode of innovation by academic
institutions.
But in the
current wording of the proposal this will only apply to ATMP's with a marketing
approval. This leaves the ATMPs for high unmet medical needs that have very
limited commercial interest and are being developed in academic research, at a
significant disadvantage. Getting a marketing approval for an ATMP for an
ultra-rare disease is both costly and takes time, if possible at all. For
paediatric cancers and other ultra-rare diseases, the market-based system for
medical innovation does not work. The revision of the EU pharmaceutical
legislation is a rare opportunity to address this.
Enabling
decentralized manufacturing for academically developed ATMPs without a
marketing authorization would help alleviating the gap in access to medicine in
paediatric cancer and other ultra-rare diseases, where the current market-based
paradigm of medical development does not suffice.
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| | | | Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or European Research Executive Agency. Neither the European Union nor the granting authority can be held responsible for them. | | | | | | | |
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|  | | | | Springer Nature Link, Published 10 April 2025. | | | | |
| Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union | |
| | | | ATMPs represent an innovative therapeutic approach with the potential to impact the treatment of rare diseases significantly. Despite the centralised authorisation in the European Union, their market launch differs across Member States and the availability of ATMPs significantly varies across Europe. Availability is essential, interconnected, and shape patients' access to these innovative therapies. This research aimed to describe the ATMP market availability in Member States and explore potential influencing factors, providing insights into specific barriers beyond pricing and reimbursement policies.
Read more here.
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| | | | .png) | | | | Nature Reviews Genetics (Nat Rev Genet). Published 10 March 2025 | | | | |
| Challenges and solutions to the sustainability of gene and cell therapies | | | | |
| The promise of gene and cell therapy has become a clinical reality for several devastating diseases. However, major hurdles constrain their economic sustainability and endanger their survival on the drug market, especially those for rare diseases. The economic sustainability of these advanced medicines is challenged by many factors that constrain their broader development and access to patients. These challenges have already led to the withdrawal from the market of some highly effective GCTs aimed at rare diseases, which were heralded among the first transforming medicines of the new millennium.
Read more here.
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| | | | .png) | | | | Value in Health, Volume 27, Issue 11, November 2024, Pages 1497-1506 | | | | |
| Policy Recommendations for Outcomes-Based Agreements in Europe | | | | |
| The challenges around making reimbursement decisions for ATMPs, including high upfront costs and uncertainties around clinical and cost-effectiveness. Managed entry agreements (MEAs) offer one approach to this issue, providing access to promising new treatments while managing uncertainty and risk through special arrangements between industry and payers. Outcomes-based agreements (OBAs) are one type of MEA that link reimbursement decisions to health and cost outcomes, with the aim of reducing the risk to payers. This study aimed to develop policy recommendations to support the acceptability and implementation of OBAs in Europe.
Read more here. | | | | | | | |
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Facilitating multinational trials | | | | |
| Regina Demlova, Director of the CREATIC and Director of CZECRIN, has been elected as the new Head of the Network Committee at ECRIN-ERIC (European Clinical Research Infrastructure Network) | | | | |
| Fostering cross-border collaboration and innovation in clinical research is instrumental to the continued development of ATMP's and in improving access to them for patients across Europe. As the director of CREATIC, Regina Demlova and her team has worked intensively with the advancement of clinical research in personalized treatment and gene therapy development.
"The appointment reflects the alignment of interests between ECRIN-ERIC and CREATIC and will hopefully improve European collaboration and streghthen the voice of the ATMP community" says Regina Demlova.
Read more here. | | | | |
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ECRIN is a European research infrastructure that facilitates researchers to set up and conduct multinational clinical trials in Europe, by linking with national European Correspondents, the national networks of clinical trial units, and through their services and tools. | | | | |
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'hustler,' 'hipster,' or 'hacker'? | | | | |
| The MUNI Health Innovation Days 2025 student competitionfocused on integrating advanced technologies for better coordination of healthcare in the Czech Republic | | | | |
| Over two days, with the support of 19 experts from fields including healthcare and technology, teams worked on projects aimed at improving the efficient sharing of information between hospitals and enhancing communication between doctors and patients.
This IDEATON was organized by the CREATIC Centre of Excellence at the Faculty of Medicine, Masaryk University, in collaboration with HEPII at the Faculty of Economics and Administration, Masaryk University, DEX Innovation Centre, and Roche.
"This year's event was particularly valuable due to its focus on innovative healthcare technologies, especially those improving communication between patients and doctors, as well as among medical professionals," said Associate Professor MUDr. Regina Demlová, Ph.D., Director of the Central European Centre for Advanced Therapies CREATIC.
Read more about the event here.
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| | | | | Rare Disease Day 2025 in Brno | |
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Rare Disease Day is observed worldwide every year on 28 February– and every fourth year on the 29th. In Brno, Rare Disease Day is organised annually by the Institute of Medical Genetics and Genomics of the Faculty of Medicine at Masaryk University and the University Hospital Brno. This year, it took place on 24 February, where experts, patient organizations, and the public came together to share insights and experiences.
Why does awareness matter? Rare diseases are often difficult to diagnose and treat. By exchanging knowledge and fostering collaboration among specialists, we can improve patient care and enhance their quality of life.
Find out more here. | | | | | | | |
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| | On November 22, 2024, The CREATIC team held the first CREATIC LabtoP talk about bringing ATMPs from lab to patients. You can now listen to the podcast from the webinar and learn more about bringing ATMPs from lab to patients, with focus on the Hospital Exemption as a pathway.
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| | Member State Coordination Group on Health
Technology Assessment explain the process for joint clinical assessments and
joint scientific consultations. The webinar is targeted at representatives of
health technology developers of medicinal products. | | | | |
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| | Stakeholder event focused on the implementation
of the European Union's Health Technology Assessment (HTA) Regulation. | | | | |
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| | A one-hour Conversations on Cancer public
panel discussion, to examine an array of challenging decisions faced by members
of the pediatric oncology community.
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| | A multi-stakeholder meeting to discuss how
innovation in the development of Advanced Therapy Medicinal Products (ATMPs)
can be translated into effective and safe therapies at the point of care in
Europe.
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| | A symposium that showcases how the
work of Committee for Advanced Therapies (CAT) has contributed to the
regulatory framework for ATMPs in Europe, its adaptation to the challenges
posed by novel classes of ATMPs and how CAT has contributed to the approval of
ATMPs.
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